Amyotrophic Lateral Sclerosis Research Program Therapeutic Idea Award | Apply Online

FAQs: ALSRP Therapeutic Idea Award (W81XWH-16-ALSRP-TIA)

1. What is the ALSRP Therapeutic Idea Award?

The Amyotrophic Lateral Sclerosis Research Program (ALSRP) Therapeutic Idea Award is a Department of Defense (DoD) funding opportunity intended to push genuinely new, early-stage concepts that could lead to drugs or other treatments for ALS. It is designed to support the front end of the therapeutic pipeline, where therapeutic ideas are generated and tested before they reach advanced preclinical development.

2. What is the main goal of this funding opportunity?

The main goal is to enable early, discovery-stage work that helps identify, define, or improve potential therapeutic interventions for ALS. The program prioritizes projects that move a therapeutic concept forward, rather than projects that primarily explain ALS biology without a direct therapeutic discovery purpose.

3. What kinds of projects are a good fit for this award?

Projects that fit well include those focused on drug or treatment discovery that are exploratory but supported by a strong scientific rationale. Examples described in the opportunity include developing, modifying, or validating preclinical model systems for evaluating therapeutic candidates, and using high-throughput screening approaches to identify or evaluate lead compounds for ALS.

4. Is this award intended for advanced preclinical development?

No. This award is explicitly focused on the discovery and early testing phase. Applicants whose work is beyond discovery and seeks support for advanced preclinical development are directed to the ALSRP Therapeutic Development Award (W81XWH-16-ALSRP-TDA), which is positioned for later-stage work.

5. Are projects focused mainly on ALS disease mechanisms allowed?

Projects centered primarily on ALS pathophysiology (for example, studies aimed mainly at describing disease mechanisms without a direct therapeutic discovery angle) are described as outside the intended scope of this award.

6. What does the program mean by "therapeutic pipeline front end"?

It refers to the stage where therapeutic ideas are conceived, refined, and initially tested, including early work to identify potential interventions and evaluate them in preclinical systems. The emphasis is on generating evidence that a therapeutic concept is worth advancing, rather than carrying out later-stage, advanced preclinical development.

7. Is high-throughput screening (HTS) specifically encouraged?

Yes. The announcement highlights high-throughput screening as a good fit, particularly screens designed to identify or evaluate lead compounds for ALS.

8. Does the award support development or validation of preclinical models?

Yes. The opportunity calls out interest in developing, modifying, or validating preclinical model systems that can be used to evaluate therapeutic candidates.

9. What does the opportunity say about "target engagement"?

The program encourages applicants to include methods to show whether a therapeutic candidate is doing what it is supposed to do in a biological system. This includes measuring target binding or more proximal downstream effects, commonly described as target engagement.

10. What does the opportunity say about selectivity and unintended targets?

The award favors projects that begin assessing whether a candidate affects related but unintended targets (selectivity). This is described as part of establishing early evidence about mechanism, specificity, and early risk signals, not just identifying an active compound or approach.

11. Is a clear hypothesis required?

Yes. Every proposal is expected to be driven by a clear, testable hypothesis and supported by a solid scientific argument for why the idea should work.

12. How "translational" does the project need to be?

Even though the work is early-stage, it needs to be translationally oriented. The application should make a convincing case for how the approach could eventually improve ALS treatment or introduce a novel treatment modality, even if a clinical result is not expected during the award period.

13. What are the major review priorities mentioned?

Innovation and impact are described as core review drivers. The award is designed for ideas that challenge existing assumptions, introduce new concepts or technologies, or otherwise take a creative approach that could open new therapeutic possibilities for ALS. Reviewers are looking for a plausible path to meaningful advancement toward treatments, not small incremental progress.

14. Does impact need to be immediate?

No. The opportunity notes that impact can be near-term or long-term, but it must be substantial. The application must explain the pathway by which the work could translate into clinical impact for people living with ALS or those at risk.

15. Are preliminary data required?

The announcement indicates that preliminary data are not the main currency for this mechanism. Relying heavily on preliminary data runs counter to the intent of enabling high-risk, high-reward ideas at an early stage. Preliminary data are not prohibited, but proposals are expected to stand on the logic of the hypothesis, the novelty of the concept, and the credibility of the experimental plan.

16. Is collaboration encouraged?

Yes. Collaboration is strongly encouraged, with a preference for meaningful, productive partnerships. The program explicitly suggests collaboration with industry partners and/or established ALS researchers when appropriate, reflecting the translational emphasis of the award.

17. Who administers this opportunity?

It is a discretionary opportunity administered by DOD-AMRAA.

18. What types of funding instruments are used?

Support is offered through cooperative agreements and/or grants.

19. What is the CFDA number associated with this opportunity?

The opportunity is listed under CFDA 12.420.

20. Who is eligible to apply?

Eligibility is listed as unrestricted, meaning it is open to any type of entity, subject to any additional eligibility clarifications in the full announcement.

21. When was the opportunity posted and when did it close?

The opportunity was posted March 1, 2016, and the closing date was July 21, 2016.

22. About how many awards were anticipated?

The announcement anticipated making about four awards.

23. What is the funding opportunity number for this award?

The funding opportunity number is W81XWH-16-ALSRP-TIA.

24. What should applicants do if their project is later-stage?

If the project is beyond the discovery stage and seeks support for advanced preclinical development, applicants are directed to the ALSRP Therapeutic Development Award (W81XWH-16-ALSRP-TDA).

25. What distinguishes a strong application under this mechanism?

Based on the description provided, a strong application would present a novel therapeutic idea grounded in a strong scientific rationale, framed by a clear and testable hypothesis, and supported by a credible experimental plan. It would be clearly aimed at therapeutic discovery (not primarily disease mechanism description), and would ideally include early indicators of mechanism-related evidence such as target engagement and selectivity considerations. The application would also clearly describe a pathway to meaningful clinical impact, even if that impact is not expected within the award period.